In a research report published in the April 2010 print issue of FASEB Journal, scientists describe how they used a non-viral, synthetic nanoparticle carrier to improve and save the sight of mice with retinitis pigmentosa. They discovered that mice receiving the nanoparticle gene therapy showed significant signs of healing. These mice had structural improvement in their retinas, as well as functional vision improvements which lasted throughout the duration of the study. The mice that received the gene alone or saline continued to lose their vision. The nanoparticles were safe and well-tolerated with no adverse effects.
“We hope the results of our study will be instrumental in generating a cure for the debilitating blindness associated with retinitis pigmentosa and other inherited and acquired retinal diseases,” said Muna I. Naash, Ph.D., a researcher involved in the work from the Department of Cell Biology at the University of Oklahoma Health Sciences Center in Oklahoma City. “Compacted DNA nanoparticles are an exciting treatment strategy for these diseases and we look forward to exciting new developments.”
Press release from the Federation of American Societies for Experimental Biology : Making the blind see: Gene therapy restores vision in mice…
Abstract: Gene delivery to mitotic and postmitotic photoreceptors via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa The FASEB Journal. 2010;24:1178-1191.
Image credit: National Eye Institute, National Institutes of Health
NOTE: We welcome Aaron Rulseh, M.D. to Medgadget editorial board. Aaron is a radiology resident at Na Homolce Hospital and a Ph.D. candidate at Univerzita Karlova in Prague, Czech Republic.